Latest New Medical Therapy Trial Results

Following are the most recent results for new drug therapies currently in clinical trials worldwide. Results are also searchable by therapeutic area beginning with the most recent updates.

Week of October 16, 2017

Eli Lilly reported that interim results from the double-blind, placebo-controlled, phase III MONARCH 3 study evaluating Verzenio (abemaciclib) in combination with a nonsteroidal aromatase inhibitor (NSAI) (anastrozole or letrozole) for progression-free survival (PFS) and objective response rate (ORR) compared to an NSAI alone in women with hormone-receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer. A total of 493 patients were randomized 2:1 to receive 150mg of abemaciclib or placebo orally twice a day, without interruption, given in combination with either 1mg of anastrozole or 2.5mg of letrozole once daily until disease progression or unacceptable toxicity. MONARCH 3 met a rigorous threshold for demonstrating efficacy at the time of pre-planned interim analysis with a 46% reduction in the risk of progression or death in patients receiving initial therapy for metastatic disease. The median PFS for abemaciclib in combination with an NSAI was not reached (i.e., the disease had not progressed significantly), compared to 14.7 months in the placebo arm (HR: 0.54; 95% CI: 0.41-0.72; p=0.000021). These results are supported by an improvement in response rate, with a 59.2% ORR in patients with measurable disease, including five patients (1.5%) achieving a complete response. Median duration of response (DoR) was not reached in the abemaciclib-plus-NSAI arm. In MONARCH 3, abemaciclib in combination with an NSAI was generally well-tolerated.

Janssen Pharmaceutical announced results from the pivotal phase III EMERALD study. The study demonstrated that switching to the investigational single-tablet regimen (STR) containing darunavir 800mg, cobicistat 150mg, emtricitabine 200mg and tenofovir alafenamide 10mg (D/C/F/TAF) was non-inferior to continuing treatment with a boosted protease inhibitor (PI) plus emtricitabine and tenofovir disoproxil fumarate in human immunodeficiency virus type 1 (HIV-1) positive, virologically suppressed adults. The study was a randomized (2:1), open-label, international, multicenter, parallel-group, non-inferiority, 48-week study in adult HIV-1 infected patients who are virologically suppressed (viral load [VL] <50c/mL for two months and had no more than one VL 50c/mL and <200 c/mL allowed within 12 months before screening). 1,141 patients were randomized and treated as follows: D/C/F/TAF (n=763); control (n=378). Inclusion criteria to be enrolled in the trial included absence of history of virologic failure on darunavir, and if historical genotype was available, absence of darunavir RAMs. Through 48 weeks, cumulative virologic rebound was 2.5% (D/C/F/TAF, n=19) vs. 2.1% (control, n=8) with 12/19 in D/C/F/TAF and 4/8 in the control group re-suppressed (<50 c/mL) by the end of the evaluation period. Additionally, at week 48, virologic suppression was 94.9% (D/C/F/TAF) and 93.7% (control), and virologic failure occurred in 0.8% and 0.5%, respectively, with no discontinuations for virologic failure and no observed RAMs to any study drug through 48 weeks. An NDA was filed on September 22, 2017, to the FDA.

Neurim Pharmaceuticals presented results of the pivotal placebo-controlled phase III study of pediatric prolonged-release melatonin formulation (PedPRM) in Autism Spectrum Disorder (ASD) children (age 2-18 years) who suffer from insomnia. The phase III study was a randomized, double-blind, parallel group, multicenter (EU and U.S.) study in children with ASD or neurogenetic diseases and sleep disorders. One hundred and twenty-five patients who had not shown improvement after standard sleep behavioral intervention, received two weeks placebo run-in, and were then randomized to PedPRM (2mg with optional increase to 5mg) or placebo in the evening, for 13 weeks. Completers received PedPRM open-label for additional 13 weeks. The study’s primary endpoint showed a clinically meaningful and statistically significant increase in total sleep time (p=0.034). Participants slept on average 57.5 minutes longer at night with PedPRM compared to 9.14 with placebo. Sleep latency (SL) decreased by 39.6 minutes on average with PedPRM compared to 12.5 with placebo (p=0.011) without causing earlier wakeup time. Sleep maintenance, parents’ quality of life and satisfaction of child’s sleep also improved significantly. The beneficial effects of PedPRM were maintained over the long term. Furthermore, compliance with the age-appropriate PedPRM formulation was good and it had a favorable safety profile over the short and long term period.

Recro Pharma reported results from a phase III study of intravenous (IV) meloxicam 30mg for the treatment of acute postoperative pain following abdominoplasty surgery. In the multicenter, randomized, double-blind, placebo-controlled clinical trial, 219 patients were enrolled and randomly assigned to receive a postoperative regimen of IV meloxicam 30mg (bolus injection) or placebo in a 1:1 ratio, once every 24 hours for up to three doses. For the study’s primary endpoint, there was a statistically significant difference in Summed Pain Intensity Difference (SPID) over the first 24 hours (SPID24) favoring IV meloxicam 30mg over placebo (p=0.0145). The study also achieved statistical significance for 10 secondary endpoints. IV meloxicam 30mg demonstrated statistically significant differences in SPID12 (p=0.0434), SPID48 (p=0.0040), SPID12-24 (p=0.0115) and SPID36-38 (p=0.0119). Statistically significant differences favoring IV meloxicam 30mg over placebo were also achieved for the number of rescue doses utilized per subject in each assessed study interval (Hour 0-24, Hour 24-48 and Hour 0-48; p<0.05). Kaplan-Meier estimates of median time to perceptible pain relief were significantly shorter for the IV meloxicam 30mg arm vs. placebo (0.76 vs. 1.28 hours, p=0.005). The results, as well as results from four phase II trials and other safety studies, comprised the NDA package that was accepted for review by the FDA in September 2017.